BowTiedBiotech 🧪🔬🧬 Profile picture
Apr 2, 2023 10 tweets 5 min read Read on X
1/ The future of #CANCER THERAPEUTICS is looking brighter than ever with innovative treatments like CAR-T, which involves engineering a patient's immune cells to target and destroy cancer.

A thread highlighting the emerging therapeutic platforms we are most excited about 👇
2/ Another exciting development is the use of IMMUNE CHECKPOINTS, which can help boost the immune response against cancer by blocking proteins that prevent the immune system from attacking cancer cells.
3/ CD3 BI-FUNCTIONAL ANTIBODIES are also a promising area of research, these drugs can target both cancer cells and T-cells, which can help enhance the immune response against cancer.
4/ ADCs, or antibody-drug conjugates, are another exciting area of cancer therapeutics, which involves using antibodies to deliver targeted chemotherapy drugs directly to cancer cells.
5/ Synthetic lethality and DNA Damage Response (DDR), are also important areas of cancer research, as they involve targeting the underlying mechanisms that allow cancer cells to survive and grow.
6/ OFF THE SHELF CAR-T and TCR-T treatments, both ex-vivo and in-vivo, are also showing promise, as they involve engineering T-cells to recognize and attack cancer cells by targeting specific proteins on the surface of cancer cells.
7/ Degraders are another innovative approach to cancer therapy, which involves using small molecules to target and degrade specific proteins that are essential for cancer cell survival.
8/ Cancer vaccines are also a promising area of research, as they involve training the immune system to recognize and attack cancer cells, much like a traditional vaccine trains the immune system to recognize and attack a virus.
9/ Myeloid checkpoints are also an exciting area of cancer research, as they involve targeting specific immune cells in the tumor microenvironment that can promote cancer growth and survival.
10/ By combining these approaches and developing new ones, we may one day be able to effectively cure many types of cancer and improve the lives of millions of people around the world. #CancerTherapeutics #InnovativeTreatments #FutureOfCancerTreatment

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More from @BowTiedBiotech

Apr 15
🧮 A “Can’t Lose” Biotech Trade

$MURA was a broken biotech with:

🔹Market cap: $21M
🔹Cash (as of 12/31): $110M
🔹Pro forma Q1 burn + severance: est. $70M-$75M remaining

And ONE major catalyst - their lead program, nemvaleukin alfa, readout.

Now that failed, here’s why the setup still works.

And most important - how the stock is UP 150%

[1/]Image
🎯 The Bullish Binary Setup

You were betting on one last data readout:

👉🏼 If it hit, market cap could go 5x-10x

👉🏼 If it missed, they had enough cash to shut it down and return capital

So you’re long a free call option on success, backed by tangible net cash.

That’s rare.

[2/]
📉 What Happens When Biotech Fails

Usually:
❌ Pipeline pivot with more burn
❌ Management clings to job security
❌ Cash erodes before value can be realized

Mural did the opposite:
✅ Stopped development
✅ Fired 90% of staff
✅ Exploring asset sale or cash return

This is optimal post-failure behavior.

[3/]
Read 8 tweets
Apr 8
1/ Biohybrid robots are here.

MIT just engineered artificial muscle that flexes in multiple directions - like your iris.

But zoom out:

What happens when you combine:
🧠 LLM-based synthetic cognition
🦾 Engineered muscle tissue
🧬 Biological self-repair

Welcome to the cyborg frontier.

Let’s dive in 🧵
2/ Muscle powers movement 💪🏼

But natural muscle isn’t one-directional - your iris, trachea, limbs use complex fiber orientations to control motion.

Yet lab-grown muscle?
Until now: one direction only.
Limited robots to twitching or crawling.
3/ 🧩 MIT engineers changed that with STAMP: Simple Templating of Actuators via Micro-topographical Patterning

A cheap, reusable, 3D-printed stamp that creates cell-scale grooves in soft hydrogels.

🧫 Seed with myoblasts
💡 Stimulate with light
⚙️ Watch them contract in multiple directionsImage
Read 13 tweets
Mar 30
Big win for $LLY in the Lp(a) race

💉EFFICACY: Lepodisiran drops Lp(a) by ~94% with just 1-2 injections

🏃‍♀️SAFETY: Effect still 74% below baseline at 1.5 years

But the real unlock?
🏆CV outcomes in 2026

[1/]Image
What is lepodisiran?

A small interfering RNA (siRNA) therapy that:

🔹Targets apo(a) production
🔹Delivered via subQ injection
🔹Silences Lp(a) at the RNA level
🔹Long-acting by design

✅ Modality: siRNA (RNAi)
❌ Not an ASO like $NVS / $IONS
⏱️ Dosing: potentially annual or less

[2/]
Phase 2 ALPACA data highlights:

400 mg dose (2 shots, Day 0/ 180):

👉🏼~94.8% Lp(a) reduction at 1 year
👉🏼~74.2% reduction still at 1.5 years
👉🏼ApoB: ~14% reduction
👉🏼No serious AE linked to drug
👉🏼No withdrawals due to treatment

This isn’t just potent - it’s durable.

[3/] Image
Read 10 tweets
Mar 6
Editas In Vivo Bet: HSC Editing 🔬🧬

Editas ($EDIT) is making a big bet on in vivo gene editing in 2025.

The company is shifting focus away from ex vivo approaches to unlock new possibilities in hematopoietic stem cells (HSCs) and liver-targeted therapies.

Here’s what’s coming. 👇Image
2/ Financial Position 💰

EDIT has $270M in cash, giving them runway into Q2 2027.

The in vivo pipeline is early, but the company plans to declare two clinical candidates by mid-2025:

1️⃣ HSC-targeted therapy for sickle cell disease (SCD) & beta thalassemia

2️⃣ Liver-targeted therapy for an undisclosed indicationImage
3/ HSC Editing for SCD & Beta-Thal

🚀 In non-human primates (NHPs), a single dose of LNP2 led to:

🔹 ~17% HBG1/2 editing in HSCs (potentially curative levels)

🔹 10% editing in mice at Day 4

If sustained, this could be a major breakthrough. More NHP data is expected mid-2025. Image
Read 10 tweets
Feb 12
1/ THREAD: Where do mRNA vaccines go after injection?

Lipid nanoparticles (LNPs) are the workhorses of mRNA vaccines, but how do they move through the body?

New PET-CT imaging in rhesus macaques gives us answers. 🧵👇 Image
2/ Tracking LNPs in real time 📡

Researchers used radiolabeled LNPs to follow vaccine movement in living primates.

PET-CT imaging at 0, 4, and 24 hours showed LNPs don’t just stay at the injection site—they move fast. Image
3/ First stop: the muscle 💪

Immediately after injection, LNPs spread through the muscle.
Read 11 tweets
Dec 2, 2023
1/ 🧠💻AI in Biopharma
Case Study: Roche

We will get more into this in the coming weeks in a Sunday newsletter. Sunday's are where we discuss topics related to emerging biopharma strategy.

But follow this thread for some super cool real life use cases from Roche 🧵👇
Image
2/ This use case caught our eye- using AI to develop clinical biomarkers for development.

That is an excellent idea! Have not seen much of this in action.

Image
Image
Image
3/ Now for the fun stuff - R&D!
We remain skeptical, but cannot lie there are some really cool things happening at Roche AI in R&D

4 Use Cases

1. Indication adjacency analysis
2. Growth conditions for screens
3. Bi/multi-fx targeting suggestions
4. Tox prediction Image
Read 12 tweets

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